Long-Term Follow-up Study of ADVM-043

NCT03804021 | Completed Results FDA Drug

• 1 other identifier: ADVM-043-03
• Study Type: observational
• Target: 6
• Locations: 1 country
• Sites: 2

Brief Summary

ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 (NCT02168686) multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.

Conditions

Alpha-1 Antitrypsin Deficiency

Keywords

Alpha-1 Antitrypsin Deficiency; alpha1-antitrypsin deficiency; alpha 1-antitrypsin deficiency; alpha1AT; A1AT; ADVM-043; AAVrh.10halpha1AT; AAVrh.10hA1AT; Gene transfer vector; Gene therapy; Lung disease; Emphysema; COPD; ADVANCE study; Long Term Follow Up

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043

  All adverse events of special interest and all serious adverse events related to ADVM-043

  2 years from start of long-term follow-up study ADVM-043-03 (NCT03804021) (2 years from completion of the parent study (ADVM-043-01, NCT02168686)

Study Arms (1)

Roll over

Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)

Genetic: No Intervention

Interventions

No Intervention GENETIC

Subjects who received ADVM-043 in a parent study (ADVANCE; NCT02168686)

Roll over

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients who received ADVM-043 in a treatment protocol

You may qualify if:

• The subject has A1AT deficiency and has previously received ADVM-043 gene therapy

You may not qualify if:

• The subject is unwilling or unable to participate in all required study evaluations in the long-term follow-up protocol
• The subject is participating in another investigational treatment protocol
• The subject is unable to understand the purpose and risks of the study or cannot provide a signed and dated informed consent form (ICF)

Sponsors & Collaborators

• Adverum Biotechnologies, Inc.: lead

Study Sites (2)

University of Florida

Gainesville, Florida, 32610, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Related Publications (1)

• Remih K, Amzou S, Strnad P. Alpha1-antitrypsin deficiency: New therapies on the horizon. Curr Opin Pharmacol. 2021 Aug;59:149-156. doi: 10.1016/j.coph.2021.06.001. Epub 2021 Jul 10.

  PMID: 34256305 DERIVED

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency; Lung Diseases; Emphysema; Pulmonary Disease, Chronic Obstructive

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Lung Diseases, Obstructive; Chronic Disease; Disease Attributes

Results Point of Contact

• Title: Chief Development Officer
• Organization: Adverum Biotechnology, Inc.

sseyedkazemi@adverum.com

650-649-1413

Study Officials

• Charlton Strange, MD

  Medical University of South Carolina, Charleston, SC, USA

  PRINCIPAL INVESTIGATOR

• Mark Brantly, MD

  University of Florida, Gainesville, FL, USA

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 11, 2019
• First Posted: January 15, 2019
• Study Start: December 27, 2018
• Primary Completion: December 6, 2021
• Study Completion: December 6, 2021
• Last Updated: September 20, 2024
• Results First Posted: September 20, 2024

Record last verified: 2023-09

Locations

US (2)